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The FDA has finalized changes to rules implementing the Orphan Drug Act. Under the new rules, "orphan subset" means a drug is appropriate for use in a subset of the population but not in the broader population. The agency also clarified that a drug may qualify for incentives even if it could be used to treat multiple patient populations, as long as each one is less than 200,000 patients.

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Access to antiretroviral therapy increased 6% among HIV-positive patients from 2009 to 2013 in the US while rates of sustained viral suppression rose 17%, according to a CDC analysis. In 2013, 94% of people getting care for HIV received antiretroviral therapy and the proportion of patients with viral suppression was 80%.

Agios Pharmaceuticals reports that the use of its AG-348 drug candidate to treat patients with anemia caused by pyruvate kinase deficiency resulted in a "rapid and sustained" increase of hemoglobin in nine of 18 trial participants. A 50-patient midstage trial is continuing.

Sage Bionetworks and DREAM, along with the NIH's National Center for Advancing Translational Sciences, the University of North Carolina and the National Institute of Environmental Health Sciences, have launched the Toxicogenetics Challenge that will use crowdsourcing to develop models that accurately predict individual and population responses to compound exposure based on genomics. Sage Bionetworks and DREAM also launched two other open source challenges: one to infer the signaling networks in breast cancer cell lines, and another to infer kinetic parameters underlying biological processes in whole cells.

The FDA rejected Johnson & Johnson's application for approval of
the leukemia drug Tipifarnib, but the company said it is committed to
developing the drug and is reviewing the FDA's concerns before deciding
the next move. The agency previously had granted "fast-track" review status to the
drug because of its potential and also designated it as an "orphan
drug."

Fabrazyme, a new drug for the rare, life-threatening Fabry's disease, has received approval after an expedited review process. It received a speedy review as part of the "orphan drug" policy, which allows treatments for life-threatening illnesses to get higher priority for approval. Fabrazyme was created through biotechnology by the Massachusetts-based firm Genzyme .